The pill blocks a mutation in bone marrow cells (Image: Getty)
Patients with a rare and deadly form of blood cancer have been ‘given a second chance’ after being given a ‘game changing’ new drug.The revolutionary treatment, known as Avapritinib, has now been approved by the government drug watchdog after trials showed a third of patients given a course of treatment were cleared of their disease. And almost all other patients on the new drug experienced ‘significant improvements’ in both signs and symptoms of their cancer.Avapritinib has now been recommended by the government drug regulator, the National Institute for Health and Care Excellence – NICE – for adults with the rare cancer known as advanced systemic mastocytosis. The devastating condition has a life expectancy ranging from 6 months to 3 years.
Drug is a ‘game-changer’ (Image: Getty)
There are no published figures on the numbers of patients affected in the UK but it is estimated hundreds are affected.
The once-a-day Avapritinib pill has been designed to block a mutation in the bone marrow cells – known as KIT – shown to drive the cancer.
Without treatment ASM can lead to severe symptoms including skin lesions, extreme diarrhoea, bone pain, fatigue, organ damage as well as life threatening allergic reactions due to the effect of the cancer on the immune system.
Dr Deepti Radia, a consultant haematologist at Guy’s and St Thomas’ NHS Foundation Trust, and lead investigator on the ongoing trial of the drug – PATHFINDER – said: “I was never sure I would see such a thing in my career. It is a gamechanger. Until now the quality of life and the prognosis for patients with the aggressive form of the cancer was terrible. This is the first drug which has shown we can completely clear the body from this disease. We have not seen this before in these patients. The drug has reversed organ damage, improved quality of life and significantly improved survival for these patients.”
Susan Rudland, mother of two, 60, from Woodbridge, was diagnosed with a form of the condition in early 2021. By July she said she was “asleep more than awake.” She added: “My bones ached, and I had dreadful pain, headaches and night sweats. I couldn’t even stand long enough to make a cup of tea. Even sitting up was painful.”
Eventually her body started to fail her, and she frequently collapsed.
Ms Rudland was hospitalised and prescribed Avapritinib in January 2022 which cleared her disease. “It was like night and day,” she said.
(pls keep mention) Jessica Hobart, Chair and Trustee at The UK Mastocytosis Support Group said: “It is truly fantastic news that this treatment, which could be life-changing for so many of the patients who are desperately waiting for it, has been made available in England and Wales without delay.”
She added: “For the first time we have a treatment that is not only able to significantly extend life, but also one that is tolerable so people can live their lives more fully.”